Trial Outcomes & Findings for Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis (NCT NCT04735432)
NCT ID: NCT04735432
Last Updated: 2023-02-28
Results Overview
ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
110 participants
Primary outcome timeframe
From week 0 to week 4
Results posted on
2023-02-28
Participant Flow
Participant's evaluation of eligibility was performed at screening and confirmed at randomization visit 1. The overall study duration per subject was approximately 12 weeks spanning the study periods - 2 weeks for screening, 3 weeks for treatment, and 7 weeks for follow-up.
153 patients were screened, 111 patients were randomized 1:1 to receive efgartigimod PH20 SC 1000 mg (55) or efgartigimod IV 10 mg/kg (56) once weekly for 4 administrations (4 doses on days 1, 8, 15, and 22). 110 patients started in the study (received treatment) as one participant randomized to the efgartigimod IV arm did not receive treatment due to an AE. 55 patients received study treatment in each treatment arm.
Participant milestones
| Measure |
Efgartigimod PH20 SC
Patients receiving efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with 1000 mg efgartigimod PH20 SC
|
Efgartigimod IV
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Overall Study
STARTED
|
55
|
55
|
|
Overall Study
COMPLETED
|
52
|
55
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
Reasons for withdrawal
| Measure |
Efgartigimod PH20 SC
Patients receiving efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with 1000 mg efgartigimod PH20 SC
|
Efgartigimod IV
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Overall Study
Other - due to personal reason
|
1
|
0
|
|
Overall Study
Adverse Event
|
2
|
0
|
Baseline Characteristics
Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis
Baseline characteristics by cohort
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
Total
n=110 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
43 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
80 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
12 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Age, Continuous
|
53.0 years
n=5 Participants
|
59.0 years
n=7 Participants
|
53.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
31 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
65 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
50 Participants
n=5 Participants
|
51 Participants
n=7 Participants
|
101 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From week 0 to week 4ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration).
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=52 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set)
|
-66.4 percent
Interval -68.91 to -63.86
|
-62.2 percent
Interval -64.67 to -59.72
|
SECONDARY outcome
Timeframe: From baseline to week 10Population: mITT Analysis Set: All randomized participants with a value for total IgG levels at baseline and at least 1 postbaseline timepoint.
Total IgG level percent change from baseline over time for the overall population.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 1
|
-40.1 percent
Standard Error 1.45
|
-39.6 percent
Standard Error 1.51
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 2
|
-56.9 percent
Standard Error 1.57
|
-55.1 percent
Standard Error 1.85
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 3
|
-62.2 percent
Standard Error 1.41
|
-59 percent
Standard Error 2
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 10
|
7.8 percent
Standard Error 7.78
|
-3.1 percent
Standard Error 3.82
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 4
|
-64.7 percent
Standard Error 1.95
|
-62.3 percent
Standard Error 1.24
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 5
|
-57.4 percent
Standard Error 1.7
|
-49.9 percent
Standard Error 2.17
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 6
|
-44 percent
Standard Error 2.52
|
-38.9 percent
Standard Error 2.24
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 7
|
-31.2 percent
Standard Error 4.67
|
-25.3 percent
Standard Error 2.78
|
|
Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)
Week 8
|
-8.9 percent
Standard Error 8.65
|
-14.6 percent
Standard Error 3.13
|
SECONDARY outcome
Timeframe: From baseline to week 10Percent reduction from baseline in AChR-Ab levels over time in AChR-Ab positive patients measured in mITT Analysis Set. Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=45 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=46 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 1
|
-42.5 percent
Standard Error 1.5
|
-43.7 percent
Standard Error 1.55
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 2
|
-57.4 percent
Standard Error 1.39
|
-55.1 percent
Standard Error 1.52
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 3
|
-61.8 percent
Standard Error 1.6
|
-59.2 percent
Standard Error 1.66
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 4
|
-62.2 percent
Standard Error 1.76
|
-59.6 percent
Standard Error 1.74
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 5
|
-55.3 percent
Standard Error 1.52
|
-47.2 percent
Standard Error 2.98
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 6
|
-40.4 percent
Standard Error 3.13
|
-29.9 percent
Standard Error 4.61
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 7
|
-26.7 percent
Standard Error 4.76
|
-15.8 percent
Standard Error 5.63
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 8
|
-14.5 percent
Standard Error 7.82
|
-7.1 percent
Standard Error 6.02
|
|
Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)
Week 10
|
13.5 percent
Standard Error 23.16
|
10.3 percent
Standard Error 7.85
|
SECONDARY outcome
Timeframe: Baseline to week 10Population: mITT Analysis Set: All randomized participants with a value for total IgG levels at baseline and at least 1 postbaseline timepoint.
Median (IQR) Percent Change From Baseline for the IgG Subtypes (IgG1, IgG2, IgG3, and IgG4) in the Overall Population. The highest number of patients among all weeks for the analysis is chosen for each arm. Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set)
Percent change from baseline at week 4 IgG1
|
-71 Percent change
Interval -79.3 to -56.6
|
-68.4 Percent change
Interval -73.0 to -61.5
|
|
Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set)
Percent change from baseline at week 4 IgG2
|
-65.6 Percent change
Interval -72.2 to -58.1
|
-64.5 Percent change
Interval -73.0 to -57.4
|
|
Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set)
Percent change from baseline at week 4 IgG3
|
-69.6 Percent change
Interval -78.3 to -58.4
|
-64.7 Percent change
Interval -76.9 to -59.5
|
|
Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set)
Percent change from baseline at week 4 IgG4
|
-56.4 Percent change
Interval -64.4 to -37.2
|
-55.5 Percent change
Interval -64.2 to -42.1
|
SECONDARY outcome
Timeframe: From baseline to week 10AUEC of the percent reduction from baseline total IgG per dosing interval (days 1-8, days 8-15, days 15-22, and days 22-29), days 1-29, days 1-57 and over the entire study (days 1-71). The highest number of patients among all weeks for the analysis is chosen for each arm. Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 1-8 (baseline-week 1)
|
-138.9 percent days
Standard Error 5.48
|
-139.1 percent days
Standard Error 5.67
|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 8-15 (week 1-week 2)
|
-341.9 percent days
Standard Error 9.9
|
-328.3 percent days
Standard Error 10.98
|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 15-22 (week 2-week 3)
|
-416.0 percent days
Standard Error 12.06
|
-399.8 percent days
Standard Error 14.46
|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 22-29 (week 3-week 4)
|
-447.3 percent days
Standard Error 9.24
|
-427.0 percent days
Standard Error 9.76
|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 1-29 (baseline-week 4)
|
-1332.5 percent days
Standard Error 30.78
|
-1311.6 percent days
Standard Error 26.35
|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 1-57 (baseline-week 8)
|
-2515.9 percent days
Standard Error 96.98
|
-2387.6 percent days
Standard Error 77.61
|
|
AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)
Days 1-71 (baseline-week 10)
|
-2562.9 percent days
Standard Error 171.86
|
-2500.3 percent days
Standard Error 116.10
|
SECONDARY outcome
Timeframe: From Week 1 to Week 4.Evaluation of observed predose concentration (Ctrough) (after all doses for the IV and SC treatment arms). The analysis will present data from Week 1 to Week 4. Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=49 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=51 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough
Ctrough week 1
|
18.3 μg/mL
Standard Deviation 8.05
|
16.4 μg/mL
Standard Deviation 33.0
|
|
Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough
Ctrough week 2
|
21.4 μg/mL
Standard Deviation 8.36
|
14.0 μg/mL
Standard Deviation 6.92
|
|
Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough
Ctrough week 3
|
22.5 μg/mL
Standard Deviation 9.61
|
15.2 μg/mL
Standard Deviation 8.05
|
|
Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough
Ctrough week 4
|
22.0 μg/mL
Standard Deviation 8.12
|
14.9 μg/mL
Standard Deviation 6.43
|
SECONDARY outcome
Timeframe: From Baseline to Week 3Evaluation of maximum observed concentration (Cmax) (after all doses for the IV treatment arm). The analysis will present data from Baseline to Week 3. Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=54 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Efgartigimod IV Serum Pharmacokinetic Parameter Cmax
Cmax Baseline
|
199 μg/mL
Standard Deviation 62.8
|
—
|
|
Efgartigimod IV Serum Pharmacokinetic Parameter Cmax
Cmax week 1
|
215 μg/mL
Standard Deviation 63.0
|
—
|
|
Efgartigimod IV Serum Pharmacokinetic Parameter Cmax
Cmax week 2
|
211 μg/mL
Standard Deviation 75.0
|
—
|
|
Efgartigimod IV Serum Pharmacokinetic Parameter Cmax
Cmax week 3
|
206 μg/mL
Standard Deviation 59.5
|
—
|
SECONDARY outcome
Timeframe: From baseline to week 10Incidence of antidrug antibodies (ADA) against Efgartigimod in the overall population. ADA analysis is performed with a validated ELISA in a 3-tiered approach (ADA screening analysis, confirmatory analysis and a titration assay). Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Incidence of ADA Against Efgartigimod (Safety Analysis Set)
|
34.5 Percent of patients
|
20.0 Percent of patients
|
SECONDARY outcome
Timeframe: From baseline to week 10Incidence of antibodies against rHuPH20 in the Efgartigimod PH20 SC Arm. antibody analysis is performed with a validated ELISA in a 3-tiered approach (screening analysis, confirmatory analysis and a titration assay) Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Incidence of Antibodies Against rHuPH20 in the SC Treatment Arm (Safety Analysis Set)
|
5.5 Percent of patients
|
—
|
SECONDARY outcome
Timeframe: From baseline to week 10Evaluation of incidence and severity of treatment-emergent adverse events (TEAEs) and incidence of serious AEs (SAEs). Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 AE
|
67.3 Percent of patients
|
50.9 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 SAE
|
14.5 Percent of patients
|
7.3 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Grade 3 or higher AE
|
16.4 Percent of patients
|
7.3 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 AESI
|
18.2 Percent of patients
|
16.4 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Injection site reaction (localized)
|
38.2 Percent of patients
|
1.8 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Infusion- or injection-related reaction
|
25.5 Percent of patients
|
3.6 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Fatal AE
|
0 Percent of patients
|
0 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Treatment-related AE according to PI
|
43.6 Percent of patients
|
21.8 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Procedure-related AE according to PI
|
25.5 Percent of patients
|
3.6 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 Treatment-related SAE
|
0 Percent of patients
|
0 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 AE for which the IMP was interrupted
|
1.8 Percent of patients
|
0 Percent of patients
|
|
Incidence and Severity of AEs and SAEs (Safety Analysis Set)
≥1 AE for which the IMP was discontinued
|
3.6 Percent of patients
|
0 Percent of patients
|
SECONDARY outcome
Timeframe: From baseline to week 10Evaluation of number and percentage of Myasthenia Gravis Activities of Daily Living (MG-ADL) responders in the overall population. The MG-ADL is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant's capacity to perform different activities in their daily life, including talking, chewing, swallowing, breathing, brushing their teeth, combing their hair, or getting up from a chair. The MG-ADL also assesses double vision and eyelid droop. It is a discrete quantitative variable in which the 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. A participant was considered a MG-ADL responder if he/she showed a reduction of at least 2 points from baseline on the MG-ADL score for at least 4 consecutive weeks. Descriptive statistics have been used for this secondary end point.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
MG-ADL Responders (ITT Analysis Set)
|
69.1 percent
|
69.1 percent
|
SECONDARY outcome
Timeframe: From Baseline to Week 10Population: One patient from Efgartigimod IV didn't have any post-baseline QMG assessment.
Evaluation of number and percentage of Quantitative Myasthenia Gravis (QMG) responders in the overall population (ITT Analysis Set). Descriptive statistics have been used for this secondary end point. One subject in the EFG IV arm had no post-baseline QMG assessment and thus was excluded from the denominator.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=54 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
QMG Responders (ITT Analysis Set)
|
65.5 percent
|
51.9 percent
|
SECONDARY outcome
Timeframe: From baseline to week 10Evaluation of MG-ADL Total Score Change from baseline over time for the overall population (ITT Analysis Set). Descriptive statistics have been used for this secondary end point. The MG-ADL is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant's capacity to perform different activities in their daily life, including talking, chewing, swallowing, breathing, brushing their teeth, combing their hair, or getting up from a chair. The MG-ADL also assesses double vision and eyelid droop. It is a discrete quantitative variable in which the 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. A participant was considered a MG-ADL responder if he/she showed a reduction of at least 2 points from baseline on the MG-ADL score for at least 4 consecutive weeks.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 1
|
-2.2 Total score on a scale
Standard Error 0.33
|
-2.0 Total score on a scale
Standard Error 0.30
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 2
|
-3.6 Total score on a scale
Standard Error 0.40
|
-3.2 Total score on a scale
Standard Error 0.35
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 3
|
-4.7 Total score on a scale
Standard Error 0.36
|
-4.3 Total score on a scale
Standard Error 0.33
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 4
|
-5.1 Total score on a scale
Standard Error 0.38
|
-4.7 Total score on a scale
Standard Error 0.37
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 5
|
-4.9 Total score on a scale
Standard Error 0.36
|
-4.3 Total score on a scale
Standard Error 0.41
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 6
|
-4.2 Total score on a scale
Standard Error 0.35
|
-3.7 Total score on a scale
Standard Error 0.44
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 7
|
-3.9 Total score on a scale
Standard Error 0.35
|
-3.6 Total score on a scale
Standard Error 0.44
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 8
|
-3.3 Total score on a scale
Standard Error 0.34
|
-2.9 Total score on a scale
Standard Error 0.4
|
|
Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set)
Week 10
|
-2.2 Total score on a scale
Standard Error 0.44
|
-2.1 Total score on a scale
Standard Error 0.43
|
SECONDARY outcome
Timeframe: From baseline to week 10Evaluation of QMG Total Score change from baseline over time for the overall population (ITT Analysis Set). The QMG (Quantitative Myasthenia Gravis) quantifies disease severity based on impairments of body function and structures as defined by the International Classification of Functioning, Disability and Health. The QMG consists of 13 items that assess ocular, bulbar, and limb function. Six of the 13 items are timed endurance tests measured in seconds. Each item has a possible score from 0 to 3. The total possible score is 39, where higher total scores indicate more severe impairments. It is based on qualitative testing of specific muscle groups to assess limb function. Descriptive statistics have been used for this secondary end point. A participant was considered a QMG responder if he/she showed a reduction of at least 3 points from baseline on the QMG score for at least 4 consecutive weeks.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=55 Participants
Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment.
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 Participants
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 1
|
-3 Total score
Standard Error 0.48
|
-2 Total score
Standard Error 0.44
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 2
|
-4.3 Total score
Standard Error 0.58
|
-3.4 Total score
Standard Error 0.44
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 3
|
-5.7 Total score
Standard Error 0.61
|
-4.5 Total score
Standard Error 0.5
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 4
|
-6.1 Total score
Standard Error 0.62
|
-5.2 Total score
Standard Error 0.52
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 5
|
-5.9 Total score
Standard Error 0.61
|
-5 Total score
Standard Error 0.57
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 6
|
-5.2 Total score
Standard Error 0.6
|
-5 Total score
Standard Error 0.61
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 7
|
-3.9 Total score
Standard Error 0.61
|
-4.1 Total score
Standard Error 0.55
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 8
|
-3.7 Total score
Standard Error 0.66
|
-3.3 Total score
Standard Error 0.53
|
|
Change From Baseline in QMG Score Over Time (ITT Analysis Set)
Week 10
|
-2.3 Total score
Standard Error 0.6
|
-2.8 Total score
Standard Error 0.53
|
Adverse Events
Efgartigimod PH20 SC
Serious events: 8 serious events
Other events: 37 other events
Deaths: 0 deaths
Efgartigimod IV
Serious events: 4 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Efgartigimod PH20 SC
n=55 participants at risk
Patients receiving efgartigimod PH20 subcutaneous (SC) treatment
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 participants at risk
Patients receiving efgartigimod intravenous (IV) treatment
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Injury, poisoning and procedural complications
Humerus fracture
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Cardiac disorders
Cardiac failure congestive
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Nervous system disorders
Myasthenia gravis
|
9.1%
5/55 • Number of events 5 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Nervous system disorders
Optic neuritis
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Nervous system disorders
Syncope
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Chest pain
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Reproductive system and breast disorders
Testicular cyst
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Infections and infestations
Cellulitis
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
Other adverse events
| Measure |
Efgartigimod PH20 SC
n=55 participants at risk
Patients receiving efgartigimod PH20 subcutaneous (SC) treatment
efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC
|
Efgartigimod IV
n=55 participants at risk
Patients receiving efgartigimod intravenous (IV) treatment
efgartigimod IV: Intravenous infusion of efgartigimod
|
|---|---|---|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
5.5%
3/55 • Number of events 3 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Injury, poisoning and procedural complications
Fall
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
5.5%
3/55 • Number of events 3 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Nervous system disorders
Headache
|
12.7%
7/55 • Number of events 10 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
12.7%
7/55 • Number of events 11 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Nervous system disorders
Myasthenia gravis
|
10.9%
6/55 • Number of events 8 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
1.8%
1/55 • Number of events 2 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Fatigue
|
3.6%
2/55 • Number of events 2 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
5.5%
3/55 • Number of events 3 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Injection site bruising
|
7.3%
4/55 • Number of events 4 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Injection site erythema
|
12.7%
7/55 • Number of events 7 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Injection site pain
|
5.5%
3/55 • Number of events 3 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Injection site pruritus
|
9.1%
5/55 • Number of events 5 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
General disorders
Injection site rash
|
14.5%
8/55 • Number of events 14 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
0.00%
0/55 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Gastrointestinal disorders
Diarrhoea
|
1.8%
1/55 • Number of events 5 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
5.5%
3/55 • Number of events 3 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
|
Infections and infestations
Urinary tract infection
|
1.8%
1/55 • Number of events 1 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
5.5%
3/55 • Number of events 3 • up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place